[Design and implementation of clinical trials for new TCM based on human use experience].

This paper systematically reviews the development history of clinical trials for new TCM drugs in China and identifies the main existing problems. It proposes that, under the "three-combination" regulatory review evidence system for TCM, clinical trials of new TCM drugs should leverage the advantages of human use experience, adhere to a clinical value-oriented approach, and flexibly select clinical development pathways without necessarily following the traditional phase Ⅱ and Ⅲ staging model. Based on human use experience data to clarify the clinical advantages of prescriptions, trial objectives should be established by determining clinical positioning, and risk-benefit assessments should be conducted to support subsequent clinical trial design. While adhering to the fundamental principles of clinical trials, new design methodologies should be introduced, including the use of adaptive designs to achieve seamless phase Ⅱ and Ⅲ integration; analysis of human use experience data to identify advantageous patient populations, providing a scientific basis for inclusion and exclusion criteria; clarification of the clinical therapeutic characteristics of TCM to guide the selection of appropriate efficacy and safety endpoints; and, based on human use experience data, estimation of sample size and determination of dosing regimens, treatment duration, visit schedules, and follow-up periods. In addition, clinical trial quality control and risk management plans should be developed, and "patient-centered" clinical trials should be implemented. These approaches aim to shorten the development timeline of new TCM drugs, reduce R&D costs, and improve the success rate of drug development.